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Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02954575
Recruitment Status : Completed
First Posted : November 3, 2016
Last Update Posted : April 18, 2018
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Description
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Brief Summary:
The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.

Condition or disease Intervention/treatment Phase
Severe Hemophilia A Drug: Wilate Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 55 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A
Actual Study Start Date : December 2016
Actual Primary Completion Date : March 29, 2018
Actual Study Completion Date : March 29, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arms and Interventions
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Arm Intervention/treatment
Experimental: All patients
All patients will receive Wilate for prophylactic treatment
 Drug: Wilate
Other Name: von Willebrand factor / Factor VIII (plasma derived)


Outcome Measures
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Primary Outcome Measures :
  1. Reduction of total annualized bleeding rate [ Time Frame: 6 months ]
    50% reduction of the total annualized bleeding rate (TABR) observed in the GENA-01 study, with a total of 58.1 BEs per patient per year.


Secondary Outcome Measures :
  1. Spontaneous annualized bleeding rate (SABR) [ Time Frame: 6 months ]
  2. Efficacy of Wilate in the treatment of breakthrough BEs based on the proportion of BEs successfully treated with Wilate [ Time Frame: 6 months ]
  3. Wilate consumption data (FVIII IU/kg per week per patient) for prophylaxis [ Time Frame: 6 months ]
  4. PK assessment (Area under of curve) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  5. PK assessment (In vivo half life) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  6. PK assessment (Maximum plasma concentration (Cmax))) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  7. Incremental IVR of Wilate over time (at baseline, and at 3 and 6 months of treatment) [ Time Frame: 6 months ]
  8. Association between ABO blood type and the FVIII:C half-life of Wilate [ Time Frame: 6 months ]
  9. Association between VWF:Ag concentration and the FVIII:C half-life of Wilate [ Time Frame: 6 months ]
  10. Safety and tolerability of Wilate by monitoring adverse events (AEs) throughout the study [ Time Frame: 6 months ]
  11. Immunogenicity of Wilate by testing for FVIII inhibitors [ Time Frame: 6 months ]
  12. Virus safety to be measured by the number of incidence of parvovirus B19 seroconversions between baseline and end of study [ Time Frame: 6 months ]

Other Outcome Measures:
  1. Efficacy of Wilate in surgical prophylaxis assessed by surgeon and hematologist using predefined assessment criteria [ Time Frame: 6 months ]

Eligibility Criteria
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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Severe hemophilia A (<1% FVIII:C) according to medical history
  2. Male patients aged ≥12 years
  3. Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)
  4. Immunocompetence (CD4+ count >200/µL)
  5. Good documentation of the historical bleeding rate (at least for the 6 months preceding study start)
  6. Voluntarily given, fully informed written and signed consent obtained by the patient (or parent/legal guardian in case of adolescents) before any study-related procedures are conducted

Whenever possible, the interval between the Screening Visit and the PK or Non-PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/µL for patients to be enrolled (i.e., exclusion criterion no. 4).

Exclusion Criteria:

  1. Any coagulation disorders other than hemophilia A
  2. History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory anti-bodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
  3. Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)
  4. Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
  5. Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02954575


Locations
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Bulgaria
Specialized Hospital for Active Treatment "Joan Pavel"
Sofia, Bulgaria
Hungary
National Haemophilia Centre
Budapest, Hungary
Poland
Krakowskie Centrum Medyczne
Krakow, Poland
Korczowski Bartosz Gabinet Lekarski
Rzeszow, Poland
Romania
Centrul Medical Unirea -Policlinica Enescu
Bucharest, Romania
Russian Federation
Barnaul Branch of RAMS hematology center
Barnaul, Russian Federation
Federal Scientific Hematology Center
Moscow, Russian Federation
Sponsors and Collaborators
Octapharma
Investigators
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Study Director: Cristina Solomon, MD Octapharma
More Information
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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT02954575     History of Changes
Other Study ID Numbers: WIL-27
First Posted: November 3, 2016    Key Record Dates
Last Update Posted: April 18, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
 Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants